Clinical trials: Anemia, Neonatal
Thalassemia
A Study to Determine the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of AG-348 in Adult Participants With Non-transfusion-dependent Thalassemia
Study AG348-C-010 is a multicenter study to evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of treatment with AG-348 in adult participants with non-transfusion-dependent thalassemia (NTDT).
- Trial name
- NCT03692052
- Testing
- AG-348
- Where
- 4 sites in United States, Canada and 1 more
- Volunteers
- 20 people
- Sponsor
- Agios Pharmaceuticals, Inc.
Sickle Cell Disease, Sickle Cell Crisis
The Use of Warmed Saline in Vaso-occlusive Episodes
The purpose of this study is to determine if warming the intravenous (IV) fluid given to patients with Sickle Cell Disease who are experiencing painful episodes known as Vaso-Occlusive Episodes; will decrease rates of hospital…
- Trial name
- NCT02316366
- Testing
- Astoflo Plus fluid warmer
- Volunteers
- 80 people
- Sponsor
- Nationwide Children's Hospital
Sickle Cell Disease
A Study Evaluating the Safety and Efficacy of Lovo-cel in Severe Sickle Cell Disease
This is a non-randomized, open label, multi-site, single dose, Phase 1/2 study in approximately 50 adults and adolescents with severe SCD.
- Trial name
- NCT02140554
- Testing
- lovo-cel
- Where
- 11 sites in United States
- Volunteers
- 54 people
- Sponsor
- Genetix Biotherapeutics Inc.
Sickle Cell Disease
Topical Sodium Nitrite in Sickle Cell Disease and Leg Ulcers
The investigators are conducting a Phase II prospective and placebo controlled study of a topical cream containing sodium nitrite compared to the current standard of care.
- Trial name
- NCT02863068
- Testing
- Topical Sodium Nitrite
- Where
- 3 sites in United States
- Volunteers
- 18 people
- Sponsor
- caterina P minniti
Sickle Cell Disease
Omega-3 Fatty Acids in Sickle Cell Disease
The purpose of this study is to determine the safety of a new formulation of the omega-3 fatty acids Docosahexaenoic Acid (DHA) and Eicosapentaenoic Acid (EPA) and to assess whether it decreases inflammation and inflammatory pain…
- Trial name
- NCT02947100
- Testing
- SCD-Omegatex™
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Robin E. Miller
Adult Acute Basophilic Leukemia, Adult Acute Eosinophilic Leukemia
Idarubicin, Cytarabine, and Tipifarnib in Treating Patients With Newly Diagnosed Myelodysplastic Syndromes or Acute Myeloid Leukemia
This phase I/II trial is studying the side effects and best dose of tipifarnib when given with idarubicin and cytarabine and to see how well it works in treating patients with newly diagnosed myelodysplastic syndromes or acute…
- Conditions
- Adult Acute Basophilic Leukemia, Adult Acute Eosinophilic Leukemia, Adult Acute Megakaryoblastic Leukemia (M7), Adult Acute Minimally Differentiated Myeloid Leukemia (M0)
- Trial name
- NCT00096122
- Testing
- cytarabine, idarubicin, tipifarnib
- Where
- United States
- Volunteers
- 95 people
- Sponsor
- National Cancer Institute (NCI)
Skin Ulcers, Sickle Cell Anemia
Phase II Randomized Trial:Arginine Butyrate Plus Standard Local Therapy in Patients With Refractory Sickle Cell Ulcers
OBJECTIVES: I. Compare the efficacy of local care alone vs local care plus arginine butyrate in terms of healing rate in patients with refractory sickle cell ulcers. II.
- Trial name
- NCT00004412
- Testing
- Arginine Butyrate, Standard local care dressing
- Where
- 4 sites in United States
- Volunteers
- 23 people
- Sponsor
- Susan P. Perrine
Sickle Cell Disease
Sickle Cell Disease Treatment With Arginine Therapy (STArT) Trial
The trial is designed to test intravenous (IV) arginine therapy in children with sickle cell disease (SCD) and vaso-occlusive painful episodes (VOE) to further knowledge on efficacy and safety of this orphan drug.
- Trial name
- STArT (NCT04839354)
- Testing
- L-Arginine Hydrochloride, Normal saline
- Where
- 10 sites in United States
- Volunteers
- 271 people
- Sponsor
- Claudia R. Morris
Sickle Cell Disease
Study to Assess Efficacy and Safety of VIT-2763 (Vamifeport) in Subjects With Sickle Cell Disease
The purpose of this study is to investigate the effect of VIT-2763 on markers of hemolysis (breakdown in red blood cells) in sickle cell disease (SCD).
- Trial name
- ViSionSerenity (NCT04817670)
- Testing
- VIT-2763 120 mg, VIT-2763 360 mg, VIT-2763 240 mg
- Where
- 22 sites in United States, France and 3 more
- Volunteers
- 25 people
- Sponsor
- Vifor (International) Inc.
Sickle Cell Disease, Anti-D Antibodies
RHD Genotype Matched Red Cells for Anti-D
This is a pilot study to evaluate the feasibility and safety of providing RH genotype matched D+ Red Blood Cells (RBCs) to chronically transfused patients with sickle cell disease (SCD) who type D+ but have formed anti-D and are…
- Trial name
- NCT04156906
- Testing
- D+ RH genotype matched red cell units for transfusion
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Children's Hospital of Philadelphia
Thalassemia, Sickle Cell Disease
Expanded Access of Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload
This is an open-label, non-randomized, multi-center trial designed to provide expanded access of deferasirox to patients with congenital disorders of red blood cells and chronic iron overload from blood transfusions who cannot…
- Conditions
- Thalassemia, Sickle Cell Disease, Diamond Blackfan Anemia, Myelofibrosis
- Trial name
- NCT00235391
- Testing
- Deferasirox
- Where
- 141 sites in United States, Belgium and 10 more
- Volunteers
- 1,683 people
- Sponsor
- Novartis Pharmaceuticals
Sickle Cell Disease
Study to Evaluate the Effect of GBT440 in Pediatrics With Sickle Cell Disease
This study consists of four parts, Parts A, B, C, and D. Part A is a single dose pharmacokinetic (PK) study in pediatric participants with Sickle Cell Disease ages 6 to 17 years.
- Trial name
- HOPE-KIDS (NCT02850406)
- Testing
- Voxelotor
- Where
- 20 sites in United States, Lebanon and 1 more
- Volunteers
- 147 people
- Sponsor
- Pfizer
Immunodeficiency Syndrome, Severe Aplastic Anemia
Fludarabine Phosphate, Cyclophosphamide, and Total-Body Irradiation Followed by Donor Bone Marrow Transplant and Cyclophosphamide, Mycophenolate Mofetil, Tacrolimus, and Sirolimus in Treating Patients With Primary Immunodeficiency Disorders or Noncancerous Inherited Disorders
This phase I/II trial studies the side effects of fludarabine phosphate, cyclophosphamide and total-body irradiation followed by donor bone marrow transplant and cyclophosphamide, mycophenolate mofetil, tacrolimus, and sirolimus…
- Conditions
- Immunodeficiency Syndrome, Severe Aplastic Anemia, Genetic Disorder
- Trial name
- NCT00358657
- Testing
- Allogeneic Bone Marrow Transplantation, Cyclophosphamide, Fludarabine Phosphate
- Where
- 3 sites in United States
- Volunteers
- 14 people
- Sponsor
- Fred Hutchinson Cancer Center
Anemia, Sickle Cell
An Assessment of Prasugrel on Healthy Adults and Sickle Cell Adults
The purpose of this study is to measure the exposure to prasugrel's active metabolite and the pharmacodynamic effects of prasugrel treatment in people with Sickle Cell Disease (SCD).
- Trial name
- NCT01178099
- Testing
- Prasugrel
- Where
- United Kingdom
- Volunteers
- 26 people, healthy volunteers welcome
- Sponsor
- Eli Lilly and Company
Infant, Newborn, Diseases, Infant, Extremely Low Birth Weight
Transfusion of Prematures Trial
The objective of the TOP trial is to determine whether higher hemoglobin thresholds for transfusing ELBW infants resulting in higher hemoglobin levels lead to improvement in the primary outcome of survival and rates of…
- Conditions
- Infant, Newborn, Diseases, Infant, Extremely Low Birth Weight, Infant, Small for Gestational Age, Bronchopulmonary Dysplasia (BPD)
- Trial name
- TOP (NCT01702805)
- Testing
- Liberal Cell Transfusion, Restricted red cell transfusion
- Where
- 20 sites in United States
- Volunteers
- 1,824 people
- Sponsor
- NICHD Neonatal Research Network
Chronic Pain, Depression, Anxiety
Cognitive Behavioral Therapy and Real-Time Pain Management Intervention for Sickle Cell Via Mobile Applications
The investigators are conducting a comparative effectiveness trial among adult patients with sickle cell disease (SCD) who report chronic pain (N = 350), randomized to receive either mobile phone-delivered computerized cognitive…
- Conditions
- Chronic Pain, Depression, Anxiety, Opioid Use
- Trial name
- CaRISMA (NCT04419168)
- Testing
- cCBT, m-Education
- Where
- 7 sites in United States
- Volunteers
- 359 people
- Sponsor
- University of Pittsburgh
Sickle Cell Disease
Effect of Ticagrelor vs. Placebo in the Reduction of Vaso-occlusive Crises in Pediatric Patients With Sickle Cell Disease
The purpose of the study is to Evaluate the Effect of Ticagrelor versus Placebo in Reducing the Rate of Vaso-Occlusive Crises in Paediatric Patients with Sickle Cell Disease
- Trial name
- HESTIA3 (NCT03615924)
- Testing
- Ticagrelor
- Where
- 55 sites in United States, Belgium and 14 more
- Volunteers
- 193 people
- Sponsor
- AstraZeneca
Sickle Cell Disease
Stem Cell Transplantation for Sickle Cell Anemia
This protocol will be investigating the use of stem cell transplantation, in related donors, to cure sickle cell disease. Sickle cell disease is a recessive disorder caused by a point mutation that results in the substitution of…
- Trial name
- NCT01877837
- Testing
- Alemtuzumab, Fludarabine, Melphalan
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Hackensack Meridian Health
Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child
Safety Study of Gene Modified Donor T-cells Following TCRαβ+ Depleted Stem Cell Transplant
This study will evaluate pediatric patients with malignant or non-malignant blood cell disorders who are having a blood stem cell transplant depleted of T cell receptor (TCR) alfa and beta cells that comes from a partially…
- Conditions
- Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child, Lymphoma, Non-Hodgkin, Myelodysplastic Syndrome
- Trial name
- NCT02065869
- Testing
- BPX-501 T cells, Rimiducid
- Where
- 4 sites in Italy, United Kingdom
- Volunteers
- 187 people
- Sponsor
- Bellicum Pharmaceuticals
Leukemia, Myelodysplastic Syndromes
Chemotherapy and Monoclonal Antibody Therapy in Treating Patients With Advanced Myeloid Cancer
RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Combining monoclonal antibody therapy with chemotherapy may kill more cancer cells.
- Conditions
- Leukemia, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms
- Trial name
- NCT00014495
- Testing
- filgrastim, cytarabine, bismuth Bi213 monoclonal antibody M195
- Where
- United States
- Volunteers
- 32 people
- Sponsor
- Memorial Sloan Kettering Cancer Center